Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of IONIS-HTTRx in Patients With Early Manifest Huntington's Disease

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Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of IONIS-HTTRx in Patients With Early Manifest Huntington's Disease

Official Title ICMJE

A Randomized, Double-blind, Placebo-controlled Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Multiple Ascending Doses of Intrathecally Administered ISIS 443139 in Patients With Early Manifest Huntington's Disease

Brief Summary

This study will test the safety, tolerability, pharmacokinetics and pharmacodynamics of multiple ascending doses of IONIS-HTTRx administered intrathecally to adult patients with early manifest Huntington's Disease.

Male or female, aged 25 to 65 years, inclusive, at the time of informed consent

Able and willing to meet all study requirements, including travel to Study Center and participation in all procedures and measurements at study visits

Have a trial partner who is reliable, competent and at least 18 years of age, is willing to accompany the patient to select trial visits and to be available to the Study Center by phone if needed

Able to tolerate MRI scans, blood draws and lumbar punctures

Reside within 4 hours travel of the Study Center

Key Exclusion Criteria:

Clinically significant medical condition, such as severe chorea, active suicidal ideation or any other conditions which would make the patient unsuitable for inclusion or could interfere with the patient participating in or completing the study

Recent treatment with another investigational drug, biological agent, or device

Prior treatment with an antisense oligonucleotide (including siRNA)

Any history of gene therapy or cell transplantation or any other experimental brain surgery

Presence of an implanted shunt for the drainage of CSF or an implanted CNS catheter

History of post-lumbar-puncture headache of moderate or severe intensity and/or blood patch

Malignancy within 5 years of Screening, except for basal or squamous cell carcinoma of the skin or carcinoma in situ of the cervix that has been successfully treated

Hospitalization for any major medical or surgical procedure involving general anesthesia within 12 weeks of Screening or planned during the study

Sex/Gender ICMJE

Sexes Eligible for Study:

All

Ages ICMJE

25 Years to 65 Years (Adult, Older Adult)

Accepts Healthy Volunteers ICMJE

No

Contacts ICMJE

Contact information is only displayed when the study is recruiting subjects